Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

PTC Therapeutics

Status and phase: Completed; Phase 2

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: Ataluren

Study type: Interventional

Funder types: Industry

Identifiers

NCT02819557

PTC124-GD-030-DMD

Details and patient eligibility

About

This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to <5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.

Full description

In nonsense mutation DMD (nmDMD), early start of treatment is important and necessary and, therefore, it is relevant to understand the correct and tolerable dose in this age group, particularly since ataluren is dosed by weight. This study included a 4-week screening period, a 52-week treatment period (the first 4 weeks of which included PK parameters), and a 4-week follow-up period for participants who completed the treatment period (60 weeks total duration). The objective of the extension period (treatment period after PK parameters have been completed) was to assess the long-term safety of chronic administration of ataluren in this participant population.

Enrollment: 14 patients

Sex: Male

Ages: 2 to 5 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Males ≥2 to <5 years of age
• Body weight ≥12 kg
• Diagnosis of DMD
• Nonsense mutation in at least 1 allele of the dystrophin gene

Exclusion criteria

• Participation in any other drug or device clinical investigation
• Ongoing use of prohibited concomitant medications

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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14 participants in 1 patient group

Ataluren

Experimental group

Description:

Participants will be administered ataluren orally at a dose of 10 milligrams/kilograms (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose will be provided based upon the weight of each participant, which will be assessed every 12 weeks.

Treatment:

Drug: Ataluren